FDA lifts partial clinical hold on Novartis SMA trial

Swiss drugmaker Novartis (NOVN: VX) has announced that the US Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) trials for spinal muscular atrophy (SMA) patients can go ahead.

The regulator’s decision to lift the partial clinical trial hold that was initiated in October 2019 was based on data from Novartis’ comprehensive non-clinical toxicology study in non-human primates that addressed all issues identified, including questions of dorsal root ganglia (DRG) injury following IT administration.

"We remain confident that investigational OAV-101 IT is a viable potential treatment path for older patients who often have ongoing unmet needs"Novartis therefore plans to initiate STEER, a global pivotal Phase III registration-enabling study to evaluate the clinical efficacy, safety, and tolerability of OAV-101 IT in treatment naïve patients who are between two and 18 years of age, able to sit, but have never walked. While disease progression is slower in patients with later-onset SMA, there are significant unmet needs.